First gene therapy for inherited disease–combating blindness–approved by FDA panel

Ivan, now 12, was diagnosed with LCA in 2006, a result of a genetic mutation on the CEP290 gene.

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Gene therapy, which has had a roller-coaster history of high hopes and devastating disappointments, took an important step forward Thursday.

A Food and Drug Administration advisory committee endorsed the first gene therapy for an inherited disorder — a rare condition that causes a progressive form of blindness that usually starts in childhood.

The recommendation came in a unanimous 16-0 vote after a daylong hearing that included emotional testimonials by doctors, parents of children blinded by the disease and from children and young adults helped by the treatment.

“Before surgery, my vision was dark. It was like sunglasses over my eyes while looking through a little tunnel,” 18-year-old Misty Lovelace of Kentucky told the committee. “I can honestly say my biggest dream came true when I got my sight. I would never give it up for anything. It was truly a miracle.”

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The FDA isn’t obligated to follow the recommendations of its advisory committees, but it usually does.

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Spark Therapeutics of Philadelphia, which developed the treatment, hasn’t said how much the company would charge. But the company has said it would help patients get access to the treatment.

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